(Gray News) - Time is running out on a potential cure for a 20-month-old boy’s disease that will leave him paralyzed. A $2 million price tag and his health insurance stand in the way of treatment.
Wade Hosey, of Washington, N.C., has red hair, blue eyes and dimples on his cheeks when he smiles. He loves to play like any other kid, but he spends most of his time crawling, only walking with help from his parents or by holding on to something.
Nicky Hosey, his mother, said he's never been able to stand on his own and learn how to balance.
"For the most part, when you look at him, he's a normal kid," Hosey said Tuesday. "When I'm holding him and you pass us in a grocery store, you think 'Oh, he's a cute little kid.' And then he starts to do things, and you're like 'Oh, that's kind of not normal.'"
Wade has spinal muscular atrophy, type 2. The rare genetic disease keeps his legs and core strength from developing normally, and it will continue to weaken his entire body.
Over time, it could affect his ability to move independently, swallow or breathe without a ventilator. Life expectancy for someone with SMA type 2 can range from early childhood to adulthood, depending on the severity of the patient’s condition, according to Cleveland Clinic.
About 1 in every 11,000 children are diagnosed with SMA. It had been incurable, but drugs on the market in the last three years offer Wade and others like him a chance at improved or even normal lives.
The latest treatment, Zolgensma, received approval in May from the Food and Drug Administration for use in children under the age of 2.
It is the most expensive drug in the world: $2.1 million for the one-time dose.
Wade’s neurologist at Duke University Hospital submitted an expedited claim request for the treatment, due to the child’s deteriorating condition and his narrow window to meet the FDA’s age limit. The family’s insurance company, Blue Cross Blue Shield of North Carolina, denied it.
That leaves the Hosey family with about 14 weeks to win an appeal with the insurance company, find financial assistance or come up with the money themselves.
Their GoFundMe has raised $29,000 in donations since July 28, still far short of the goal.
“I think he’s just started to notice (the disease) … this week,” Hosey said of her son. "He’s at the age to start noticing, he’s almost 2. And he picks his legs up a lot, so if he’s sitting there, or even if he’s standing there, he will grab his skin on his legs and try to pick them up.
"Or he'll grab his feet and try to move them. It's like they're just dead weight to him."
On Wednesday, the FDA announced Novartis admitted to data manipulation during initial product testing for Zolgensma, which it reported after the gene therapy was approved.
The company claimed the investigation found no issues with product safety or quality, per The Wall Street Journal. The FDA said the drug should remain on the market.
"These data do not change the agency's positive assessment of the information from the human clinical trials that were conducted as part of the development program," said the FDA in a statement.
Hosey said she still believes Zolgensma is the best option for Wade, based on the success of children who participated in the trials.
The drug is given intravenously, and it works by delivering a normal copy of the mutated gene that causes SMA to the patient’s motor neurons. The delivery method uses an adeno-associated virus, AAV9, for its ability to cross blood vessels and get into muscles, and antibodies developed as the child gets older than 2 can kill the virus and prevent the drug from working.
Novartis, the company that markets Zolgensma, was one of the most profitable pharmaceutical companies in the world, with a net income of $12.6 billion, in 2018. It explained the price of its $2 million drug by saying it is half the cost of 10 years of chronic SMA therapy.
The company offers a pay-over-time option of up to five years ($425,000/year) "to help ease possible short-term budget constraints" according to a news release. Novartis purchased AveXis, the maker of the drug, for $8.7 billion last year.
Juliana Keeping, with the advocacy group Patients for Affordable Drugs, called it outrageous that Novartis would seek approval with false information on the most expensive drug ever.
"They lied about the data and then they slapped this price tag on the drug," Keeping said. "It's unjustifiable."
Novartis representatives did not respond to requests for comment.
Blue Cross Blue Shield of North Carolina’s policy states it will only cover Zolgensma if the patient meets all six of its criteria. In its denial letter, it said it would not pay for Wade’s doctor-recommended treatment because he did not have SMA type 1, the most severe form of the disease.
The health insurance company covers nearly 3.8 million people, with profits around $700 million per year, based on a 2-year average. A spokesperson would not comment on Wade's case, citing HIPAA regulations.
Keeping said high drug prices leave patients stuck in the middle of battles between drug companies and insurers.
"With the $2.1 million price put on Zolgensma for SMA, you see insurers literally writing gene therapies out of their plan and refusing to pay for it, putting these medically vulnerable children, whose lives are already imperiled, at further risk," Keeping said.
When Wade showed no signs that he was ready to stand or walk at about 12 months, his parents began to worry.
Their fears grew worse when he still wasn't walking at 18 months, so they had genetic testing done. They had never heard of SMA before getting the diagnosis in July.
"The first thing you do, of course, is you Google it," his mom said. "And you see a kid in a wheelchair who cannot move their arms or their legs. They can't move their neck; they have a ventilator and a trach in their throat. And you lose it."
She said the feeling of hope, knowing a treatment is out there, has been mixed with depression and anger. She compared the fight to save her son to a brick wall that's not moving.
"Insurance is telling me that the doctor hasn't sent (paperwork) in, but the doctor has proof that they've sent it in," she said. "Insurance is like, "Well, we've got to go through all this.' I would not wish this on my worst enemy."
She said Wade would likely be able to walk if he gets Zolgensma, and the odds of him ever needing a respirator were slim to none. The tremors in his hands and body would slow down, and his core would strengthen.
"He's going to be in a wheelchair (without treatment) - it's a matter of if it's going to be six months or two years," she said. "But there's a possibility that he might never have to be in a wheelchair if he gets this."
The Hoseys are seeking out doctors with experience with Zolgensma for guidance and possible treatment.
With the insurance company getting between Wade’s family and their physicians, his mother said they also are checking into alternates ways to treat him. They appealed the decision by Blue Cross Blue Shield of North Carolina, and they have sought financial assistance from the drug company.
People can apply to the Novartis-AveXis to get part of the cost covered. That would leave Wade's family on the hook for around $400,000, Hosey said.
Their insurance approved Wade for another SMA drug, Spinraza. The first treatment entails multiple injections into the spine, with additional injections every four months for the rest of his life.
Hosey said her initial out-of-pocket cost for Spinraza would be $80,000. The Washington Post reported the overall cost is around $750,000 for the first year and $375,000 in each subsequent year of treatment, exceeding the price of Zolgensma within a few years.
Hosey is afraid insurance may not continue to pay for the treatment all of Wade’s life.
"These pharmaceutical companies are in control, and there's nothing we can do about it," Wade's mother said. "It's a problem that all of these politicians go up to (Washington) D.C. and they want to go fix. For some reason, they get up there and nothing gets done.
"It's a crisis. It's an epidemic, that's what it is."
UnitedHealthcare reversed two claims it previously denied in July for Zolgensma. It has also approved four others for the treatment on their first submission.
The approvals came one week after Washington Post reported on the appeals for the families who had been denied. UnitedHealth Group’s chief medical officer, Richard Migliori told The Post that the reversals were due to clinical evidence, not public pressure.
UnitedHealth allows claims on the drug for children up to 2 years old, if they match the company's own set of criteria.
Keeping said Zolgensma was one of the first gene therapies to be approved by the FDA, but there are hundreds more in the pipeline.
"The tsunami is coming with these expensive therapies," she said. "We're watching the first victims from these initial (drug) approvals, with the babies and toddlers who require Zolgensma. The (healthcare) system is going to buckle under the weight of these inexcusable price tags."